ENABLE: The First Phase 4 Observational Study for Patients with Relapsing MS Treated with Ublituximab in Real World Clinical Setting
Edward Fox1, Jackie Parker1, Karthik Bodhinathan1, Peter Sportelli1, Hari Miskin1, Carrie Hersh2
1TG Therapeutics, 2Cleveland Clinic Lou Ruvo Center for Brain Health
Objective:
To evaluate the real-world clinical effectiveness, safety, and tolerability of ublituximab.
Background:
Ublituximab, approved for treating relapsing multiple sclerosis (RMS), has demonstrated significant clinical benefits vs teriflunomide in two identical phase 3 trials, ULTIMATE I and II. These benefits continued to be observed over 5 years in open-label follow-up. Evaluating the rEal-world experieNce of patients treated with BRIUMVI® (ublituximAB-xiiy) for relapsing multiple sclerosis (RMS), in a Longitudinal rEgistry format (ENABLE) will provide valuable real-world clinical evidence on the effectiveness, safety, and tolerability of ublituximab, complementing the observed data from pivotal studies.
Design/Methods:
ENABLE (NCT06433752) is a 96-week, multicenter, phase 4 observational study to collect real-world data from ublituximab-treated patients. The primary and secondary objectives are to evaluate the effectiveness and safety and tolerability of ublituximab, respectively. Exploratory objectives include patient-reported outcomes (PROs), total infusion time, laboratory assessments, and MS outcomes.
Results:
The study aims to enroll at least 500 RMS patients across 100 centers in the US, capturing a diverse geographic and racial/ethnic population from a wide range of MS centers spanning academic hospitals and independent clinics. Enrollment is expected to take up to 24 months, and several sites have opened starting mid-2024. The primary endpoint is annualized relapse rates at week 96. The secondary endpoint is the proportion of participants experiencing adverse events in addition to documenting the incidence, severity, and type of infusion-related reactions (IRR) at each infusion. Exploratory endpoints include PROs (TSQM 1.4 and MSIS-29), infusion time (including premedication and post-infusion observation), and changes in immunoglobulins, B cell counts, MRI activity, Expanded Disability Status Scale, Symbol Digit Modalities Test (SDMT), Timed 25-foot Walk (T25-FW), and Nine-Hole Peg Test (9-HPT).
Conclusions:
ENABLE will provide valuable real-world clinical evidence on the effectiveness, safety, and tolerability of ublituximab from MS centers across the US.
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